Market Access Granted in France for Aggressive non-Hodgkin Lymphoma Treatment,
PIXUVRI® (pixantrone)
PR Newswire
SEATTLE, Aug. 19, 2013
SEATTLE, Aug. 19, 2013 /PRNewswire/ --Cell Therapeutics, Inc. (CTI) (NASDAQ
and MTA: CTIC) today reported that the Transparency Commission (Commission de
la Transparence or CT) of the French National Health Authority (Haute
Autorite de Sante or HAS) has granted market access for the medicinal product
PIXUVRI^® (pixantrone) as a monotherapy for the treatment of adult patients
with multiply relapsed or refractory aggressiveB-cell non-Hodgkin lymphoma
(patients with aggressive B-cell NHL who failed 2 or 3 prior lines of
therapy). The next and final step in France's pharmaceutical reimbursement
process is inclusion on the list of medicines approved for hospital use and
subsequent publication in the Journal Officiel in France, which CTI now
intends to pursue.
"The availability of PIXUVRI in France means physicians will be able to extend
an approved salvage regimen to those patients that fail second line therapy,"
said Gilles Salles, M.D., PhD., Professor of Medicine at the University Claude
Bernard Lyon-1 and President of LYSA, the Lymphoma Study Association.
"The data from the pivotal EXTEND study of PIXUVRI clearly indicate that this
drug is effective in heavily pretreated patients with relapsed or refractory
aggressive NHL," commented Bertrand Coiffier, M.D., Professor of Haematology
at the Department of Haematology, Hospices Civils de Lyon and the University
Claude Bernard, Lyon, France.
The CT noted that aggressive NHL is a serious disease and that there are no
standard treatments for patients with this stage of disease. All registered
pharmaceuticals in France are subjected to an evaluation process known as
Evaluation of Therapeutic Benefit (Amelioration du Service Medical Rendu, or
ASMR) by the CT, with the resulting evaluation expressed as a classification
between 1 and 6. The CT rated PIXUVRI at level 5, which allows PIXUVRI to be
included in the reimbursed drugs list for hospital use. The CT will reassess
the ASMR rating for PIXUVRI within two years.
"We are pleased to receive the CT's favorable opinion for reimbursement of
PIXUVRI and look forward to bringing this new approved therapy to patients in
France with aggressive NHL," said James A. Bianco, M.D., President and CEO of
CTI.
In May 2012, the European Commission (EC) granted conditional marketing
authorization in the European Union (E.U.) for PIXUVRI as a monotherapy for
adult patients with multiply relapsed or refractory aggressive B-cell NHL
based on the results of the EXTEND, or PIX301, pivotal randomized Phase 3
clinical trial. PIXUVRI was made available to patients in eight countries in
the European Union in the fourth quarter of 2012, and some patients in other
countries have already started to receive the treatment. Prior to the
approval of PIXUVRI in the E.U., there were no approved agents or standard of
care in this disease. The PIX301 trial was designed utilizing agents in the
comparator arm that have anti-tumor activity in relapsed disease and are
typically employed as palliative therapy for these patients.
About PIXUVRI (pixantrone)
PIXUVRI is a novel aza-anthracenedione with unique structural and
physiochemical properties. Unlike related compounds,PIXUVRI forms stable DNA
adducts and in preclinical models has superior anti-lymphoma activity compared
to related compounds. PIXUVRI was structurally designed so that it cannot
bind iron and perpetuate oxygen radical production or form a long-lived
hydroxyl metabolite -- both of which are the putative mechanisms for
anthracycline induced acute and chronic cardiotoxicity. These novel
pharmacologic properties allow PIXUVRI to be administered to patients with
near maximal lifetime exposure to anthracyclines without unacceptable rates of
cardiotoxicity.
InMay 2012, the European Commission (EC) granted conditional marketing
authorization for PIXUVRI as a monotherapy for the treatment of adult patients
with multiply relapsed or refractory aggressiveNHL. The benefit of PIXUVRI
treatment has not been established in patients when used as fifth line or
greater chemotherapy in patients who are refractory to last therapy. The
Summary of Product Characteristics (SmPC) has the full prescribing
information, including the safety and efficacy profile of PIXUVRI in the
approved indication. TheSmPCis available atwww.pixuvri.eu.
CTI is currently accruing patients into a Phase 3 trial comparing PIXUVRI and
rituximab with gemcitabine and rituximab in the setting of aggressive B-cell
NHL. PIXUVRI does not have marketing approval inthe United States.
About Non-Hodgkin Lymphoma
NHL is caused by the abnormal proliferation of lymphocytes, cells that are key
to the functioning of the immune system. It usually originates in lymph nodes
and spreads through the lymphatic system. NHL can be broadly classified into
two main forms—aggressive and indolent NHL. Aggressive NHL is a rapidly
growing form of the disease that moves into advanced stages much faster than
indolent NHL, which progresses more slowly.
According to France's Institut de Veille Sanitaire, in 2010 there were 10,800
newly diagnosed patients with NHL in 2010.^1 There are many subtypes of NHL,
but aggressive B-cell NHL is the most common and accounts for about 50 percent
of NHL cases.^2 After initial therapy for aggressive NHL with
anthracycline-based combination therapy, one-third of patients typically
develop progressive disease.^3 Approximately half of these patients are
likely to be eligible for intensive second-line treatment and stem cell
transplantation, although 50 percent are expected not to respond.^3 For those
patients who fail to respond or relapse following second-line treatment,
treatment options are limited, and usually palliative only.^3
About Conditional Marketing Authorization
Similar to accelerated approval regulations intheUnited States, conditional
marketing authorizations are granted in the E.U. to medicinal products with a
positive benefit/risk assessmentthat address unmet medical needs and whose
availability would result in a significant public health benefit. A
conditional marketing authorization is renewable annually. Under the
provisions of the conditional marketing authorization for PIXUVRI, CTI will be
required to complete a post-marketing study aimed at confirming the clinical
benefit previously observed.
The European Medicines Agency's (the EMA)Committee for Medicinal Products for
Human Usehas accepted PIX306, CTI's ongoing randomized controlled Phase 3
clinical trial, which compares PIXUVRI-rituximab to gemcitabine-rituximab in
patients who have relapsed after one to three prior regimens for aggressive
B‑cellNHLand who are not eligible for autologous stem cell transplant. As a
condition of approval, CTI has agreed to have available the PIX306 clinical
trial results byJune 2015.
About Cell Therapeutics, Inc.
CTI (NASDAQ and MTA: CTIC) is a biopharmaceutical company committed to the
development and commercialization of an integrated portfolio of oncology
products aimed at making cancer more treatable. CTI is headquartered in
Seattle, WA. For additional information and to sign up for email alerts and
get RSS feeds, please visit www.CellTherapeutics.com.
Forward-Looking Statements
This press release includes forward-looking statements within the meaning of
the Safe Harbor provisions of the Private Securities Litigation Reform Act of
1995. Such statements are subject to a number of risks and uncertainties, the
outcome of which could materially and/or adversely affect actual future
results and the trading price of CTI's securities. Such statements include,
but are not limited to, statements regarding expectations with respect to the
following: the development of CTI and its product and product candidate
portfolio; future inclusion of PIXUVRI on the list of medicines approved for
hospital use and subsequent publication in the Journal Officiel in France; the
effectiveness of PIXUVRI in heavily pretreated patients with relapsed or
refractory aggressive NHL; and CT's expected review of the ASMR rating for
PIXUVRI within two years. Risks that contribute to the uncertain nature of
the forward-looking statements include, among others, risks associated with
the biopharmaceutical industry in general and with CTI and its product and
product candidate portfolio in particular including, among others, risks
associated with the following: that CTI cannot predict or guarantee the pace
or geography of enrollment of its clinical trials, that CTI cannot predict or
guarantee the outcome of preclinical and clinical studies, that CTI may not
obtain reimbursement for PIXUVRI in certain markets in the European Union as
planned, that the conditional marketing authorization for PIXUVRI may not be
renewed, that the second Phase 3 clinical trial of pacritinib will not occur
as planned, that CTI may not obtain favorable determinations by other
regulatory, patent and administrative governmental authorities, that CTI may
experience delays in the commencement of preclinical and clinical studies,
risks related to the costs of developing, producing and selling PIXUVRI,
pacritinib, and CTI's other product candidates, and other risks, including,
without limitation, competitive factors, technological developments, costs of
developing, producing and selling PIXUVRI, that CTI's operating expenses
continue to exceed its net revenues, that CTI may not be able to sustain its
current cost controls or further reduce its operating expenses, that CTI's
average net operating burn rate may increase, that CTI will continue to need
to raise capital to fund its operating expenses, but may not be able to raise
sufficient amounts to fund its continued operation, as well as other risks
listed or described from time to time in CTI's most recent filings with the
Securities and Exchange Commission on Forms 10-K, 10-Q and 8-K. Except as
required by law, CTI does not intend to update any of the statements in this
press release upon further developments.
PIXUVRI is a registered trademark of Cell Therapeutics, Inc.
References:
1. Projected incidence and cancer mortality in France in 2010. Available at
http://www.invs.sante.fr/applications/cancers/projections2010/default.htm
2. Harris NL, et al. Ann Oncol. 1999;10(12):1419-32
3. Friedberg, ASH Education Book 2011;1:498-505
Contacts: Monique Greer
+1 206-272-4343
mgreer@ctiseattle.com
Ed Bell
+1 206.282.7100
ebell@ctiseattle.com
In Europe
CTI Life Sciences Limited, Milan Branch
Laura Villa
E: lvilla@cti-lifesciences.com
T: +39 02 89659706
http://www.celltherapeutics.com/italiano
SOURCE Cell Therapeutics, Inc.
Website: http://www.celltherapeutics.com
Website: http://www.pixuvri.eu